Gene Therapy for Chronic Granulomatous Disease in Korea

• Sponsor: Helixmith Co., Ltd. (Industry)
• Overall Status: Active, not recruiting
• CT.gov ID: NCT00778882
• Collaborator: (none)
• Enrollment: 2
• Location: 1
• Arm: 1
• Duration (Months): 189
• Patients Per Site Per Month: 0

Study Details

Study Description

Brief Summary

The purpose of this study is to evaluate the safety and efficacy of administration of autologous hematopoietic stem cells transduced with MT-gp91 retroviral vector for patients with X-linked chronic granulomatous disease.

Condition or Disease	Intervention/Treatment	Phase
Chronic Granulomatous Disease	Drug: VM106	Phase 1/Phase 2

Study Design

• Study Type: Interventional
• Actual Enrollment: 2 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: An Open-label, Uncontrolled, Single Center, Phase I/II Trial to Assess the Safety and Efficacy of Autologous Hematopoietic Stem Cells Transduced With MT-gp91 Retroviral Vector in gp91 Defective Chronic Granulomatous Disease Patients
• Study Start Date: Jan 1, 2007
• Actual Primary Completion Date: Oct 1, 2008
• Anticipated Study Completion Date: Oct 1, 2022

Arms and Interventions

Arm	Intervention/Treatment
Experimental: VM106	Drug: VM106; Autologous hematopoietic stem cells with MT-gp91 retroviral vector

Outcome Measures

Primary Outcome Measures

1. The incidence of adverse events through 1 year [1 year]

Secondary Outcome Measures

1. RCR, insertional mutagenesis, immune response against normal gp91 protein [1 year]

2. Safety and efficacy of fludarabine/busulfan conditioning [1 year]

3. Functional reconstitution of respiratory burst [1 year]

4. Presence of vector-positive cells [1 year]

Eligibility Criteria

Criteria

• Ages Eligible for Study: N/A and Older
• Sexes Eligible for Study: Male
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• gp91 defective male patients with chronic granulomatous disease: confirmed by DHR

• Weigh greater than or equal to 15 kg

• History of severe infections: more than 2 times

• Performance status: ECOG 0-2

• Patients must be free of significant functional deficits in major organs, but the following eligibility criteria may be modified in individual cases.

• Heart: a shortening fraction > 28%; QTc interval < 0.44

• Liver: total bilirubin < 2 × upper limit of normal; ALT < 3 × upper limit of normal; AST < 3 x upper limit of normal

• Kidney: creatine < 2 x normal

• Blood: WBC > 2,500/uL; platelet > 100,000/uL; hematocrit > 26%

• Written informed consent obtained from patient (or guardian if patients age < 19)

Exclusion Criteria:

• Presence of a HLA-matched sibling for stem cell donation

• Evidence or history of malignant tumor

• Presence of a severe infection

• Presence of an active tuberculosis

• Uncorrectable electrolyte, Ca, P

• Unable to comply with the protocol or to cooperate fully with the Investigator or site personnel

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Seoul National University Hospital	Seoul		Korea, Republic of	110-744

Sponsors and Collaborators

• Helixmith Co., Ltd.

Investigators

• Principal Investigator: Joong Gon Kim, MD, PhD, Seoul National University Hospital

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Helixmith Co., Ltd.
• ClinicalTrials.gov Identifier: NCT00778882
• Other Study ID Numbers: VM106-KR-01
• First Posted: Oct 24, 2008
• Last Update Posted: Oct 18, 2019
• Last Verified: Oct 1, 2019

• Keywords provided by Helixmith Co., Ltd.: Chronic Granulomatous Disease; Retroviral Vector; Gene Therapy
• Additional relevant MeSH terms: Granuloma; Granulomatous Disease, Chronic

Study Results

No Results Posted as of Oct 18, 2019